Triheptanoin - A Promising Therapy for Several Clinical Conditions

In recent days triheptanoin has gained considerable attention regarding its clinical effects in treatming inherited metabolic diseases, epilepsy, ventricular hypertrophy and myocardial glucose oxidation. Here I would like to explore a little more about this chemical.

What is triheptanoin?

Triheptanoin, sometimes also referred to as “C7 Oil,” is a triglyceride oil, as its name suggests, composed of three seven-carbon fatty acids. It is almost tasteless and flavorless. By now triheptanoin is used to treat rare metabolic disorders in USA and France. Sometimes it is also used in the cosmetic industry as an ingredient of certain cosmetic products. In some European countries, this chemical is added to butter for the purpose of tracing of the origin of the butter.

Triheptanoin could be taken as a dietary supplement and is currently under investigation as a therapy for Glut1 Deficiency. Triheptanoin is also used experimentally to treat a variety of metabolic disorders such as Fatty Acid Oxidation Disorder (FAOD), Pyruvate Carboxylase Deficiency and Carnitine Palmitoyltransferase II Deficiency, and is under investigation as a potential therapy for epilepsy and Alzheimer’s disease.

Now let’s take a look at the two major potential clinic functions of triheptanoin.

For Glut1 Deficiency

As we all know that glucose is the major source of energy for the brain, so patients with Glut1 deficiency often suffers from brain starvation for energy supply resulting from lack of transfer from blood to brain. When triheptanoin is taken as a therapy and converted into ketone bodies in the liver, they can cross the blood-brain barrier, bringing alternative source of energy for the brain. In other words, we could say that triheptanoin has an anaplerotic role. However, currently triheptanoin for treating Glut1 deficiency is still at the stage of clinic researcher and there is still a long way to go before broader use.

Except for its therapeutical efficacy in kids and adults with Glut1 deficiency, there are increasing preclinical and clinical studies indicating that triheptanoin is beneficial in other neurological and neuromuscular disorders, such as epilepsy and Alzheimer’s Disease, despite the fact that AD is primarily treated by increasing the so-called neurotransmitters such as donepezil and memantine in the brain.

According to Chu and Jiao, 2015 and other researchers as well, the brain “exhibits a deficiency in glucose metabolism during early stages of AD, which contributes to the neurodegeneration and progression of the disorder. While in AD brain glucose uptake is impaired, ketone uptake stays unaffected, based on Cunnane et al. Triheptanoin could serve as a potential therapeutic approach through supplying ketones to the brains of AD patients and thus restore the brain energy supply.